Survey and Policy

Survey

Background

Wei Zexi (1994 – April 12, 2016) was a 21-year old Chinese college student from Shaanxi who died after receiving experimental treatment for synovial sarcoma at the Second Hospital of the Beijing Armed Police Corps, which he learned of from a promoted advertisement on the Chinese search engine Baidu. Medias outlets broadly condemned the role of the hospital and Baidu in his death, and users online denounced Baidu's advertising practices. The incident was a big shock to the public’s confidence and acceptance of bio-therapy in China.

Purpose

1. Gauge public acceptance of our project.

2. Determine how doctors or other healthcare workers think about neurodegenerative diseases and any concerns about the biotherapies of neurodegenerative disease.

3. Determine how patients and their relatives think about the therapies of neurodegenerative diseases applying exosomes.

Method and Procedure

We conducted a survey through questionnaires about neurodegenerative diseases, current biotherapies. The respondents of the survey had to be doctors or other healthcare workers. The following survey with all multiple choice questions was designed and double-checked in order to reduce any potential bias.

As previously mentioned, the respondents of this survey had to be healthcare workers. After calculating the identity of them, a majority of respondents are administrative management or support staff and most of the participants are doctors. This graph shows that most people have no biological cases for treating neurodegenerative diseases around them, while there do have someone worked with some cases of treating neurodegenerative diseases by biotherapy.

After the relevant technology development in the Drug Delivery, 57% of healthcare workers could recommend a biological method to treat patients with neurodegenerative disease. That implies the great development of biological technology and potential perspective that biotherapy could apply to the treatment of neurodegenerative disease in the future. Around one-half of health care workers said that the biotherapy for neurodegenerative diseases is highly precise. In other words, traditional methods cannot be used to cure diseases in the future by the respondents’ opinion.

Most healthcare people did not sure about that biotherapy could be used in medicine on a large scale in the future, maybe because that the newly developing biotherapy is not yet mature in technology and the number of successful cases is not large enough. Gene sequencing and detection, computer-aided diagnosis and biomarker detection are known by the respondents, implying that the basic technology in biotherapy is relatively widely used in medicine.

Most health care workers have never use biodiagnostic methods in the clinic but someone, though only a minority of them, have sometimes or seldom used biodiagnostic methods. What health care know more about the new mode of Drug Delivery using exosomes is that the genetically engineered cells cause the cells to “produce” the exosomes and encapsulate the drug (or protein/RNA) in the exosomes.

About half of the healthcare workers heard about the Drug Delivery using exosomes in biotherapy while half of them didn’t hear about it. About 70% of healthcare workers have no ideas whether applying exosomes is good or not, implying that Drug Delivery using exosomes is a new method of biotherapy. Half of the healthcare workers think that the validity and high cost could be the major possible problems within exosome as Drug Delivery in biotherapy.

Integration into Project Design

In the feedback, we could draw the conclusion that most of the healthcare workers know little about the biotherapy and exosome used as Drug Delivery on the treatment of the neurodegenerative diseases. Additionally, traditional therapies and medicines are widely known by the respondents but they stated that traditional methods cannot be used in the future may be because of some inevitable drawbacks. Furthermore, they also worry a lot about biotherapy or Drug Delivery using exosomes, especially in the high price and the low validation. Overall, biotherapy has a potential perspective in the treatment of disease in the future and the Drug Delivery using exosomes could be a new valid therapy in treating neurodegenerative diseases.

Policy

We conducted a research on China's bioproducts and biomedicine evaluation policy this year. Firstly, we did a literature review about the policy, regulation, and laws regarding the interaction of biopharmaceutical industries and government in China. We interviewed Dr. Xianglei Meng, who is Pharmacy expert working as Deputy Director of the Pharmacy Department of a hospital, consulted the operation process and management system of the pharmacy in China. Beyond that, we paid a visit to a leading pharmaceutical research companies Genequantum, talked with Dr. Chong Liu and Mr. Yunpeng Zhong about the policies and laws of biopharmaceutical product approval process and clinical trials in China.

A few years ago, China's biological agent and drugs evaluation system was in an extremely immature state. The scientific and technological innovation support for pharmaceutical medical devices as not enough, and there was a gap between the quality of listed products and the international advanced level. However, after a series of reforms by the State Food and Drug Administration in recent years, the situation has clearly improved. China's drug regulators regulations are working hard to align with world standards. Innovative drugs, biological products, drugs that address major public health risks, and drugs that treat rare diseases have all been included in the priority review channel.

In our questionnaires, interviews and field research, we found the following:

(1) Despite a series of laws and regulations and related policies being declared, some of them lack detailed implementation rules and specific enforcement regulation, resulting in the lack of effective guidance for pharmaceutical companies in actual operations. For example, there are ambiguities and uncertainties in the actual operation of clinical trials’ evaluation and drug approval process and it is complicated and time-consuming.

(2) China's current regulations required that GMP can only be certified to qualified drug manufacturers and associated to products. Non-certified laboratories or pilot plants are currently considered “non-GMP”. China requires GMP certification in the first phase of clinical trials of drugs. Differently, the United States, Canada, and many other countries and regions are exempted from the requirements of GMP for Phase 1. These problems have posed a dilemma to many drug development companies: to conduct drug trials in foreign countries or to spend massively in China to work with companies that hold GMP qualifications.

(3) The public has insufficient confidence in biological therapy. According to our survey, not only ordinary people, but even some doctors are skeptical about biological treatment. After a series of events, public opinion has exerted pressure, causing the government and hospitals to adopt more cautious approaches to experimental and clinical biotherapy.

(4) Ethical supervision and biosafety supervision need to be strengthened. Due to different cultural backgrounds, the ethical review of biomedical research started late in China, leading to imperfect regulations and inefficient ethical review; many researchers lacked knowledge and correct understanding. Similarly, biosecurity is a serious challenge in China and the world. Current regulations do not effectively control the biosafety risks of the entire process of research, production, experimentation, and application.

Figure: The drug evaluation and approval process of China

Our recommendations and plans for future project improvement:

(1) In response to regulations and policies, we hope to have extensive contacts and communication with more government departments in the comprehensive human practice process in the future to understand the practical difficulties in the drug review process. Meanwhile, we can cooperate with the company and analyze the current challenge they are facing, and to provide a concrete policy advice report.

(2) We call for the addition of research ethics in basic education, undergraduate and postgraduate education to raise the awareness of public. At the same time, we hope to have more teams (especially in China) in the future of education and in social participation projects; students and the public should be guided to pay attention to the ethics and philosophy behind science.

(3) We propose to establish an effective communication mechanism to bring together various stakeholders, involving the public, drug developers, industry and government to reduce information barriers, and to refine the laws and regulations to make an effective trade-off between discretionary power and uncertainty.

(4) We suggest that China's drug approval and evaluation policy should be further integrated with the world, for example, accepting overseas clinical trial data; encouraging a series of extended experiments; focusing on the transparency of experimental animal welfare and clinical subjects; and cultivating more efficient drugs; approving and evaluate talents; considering contracting part of the work to commercial contractors.